Dr. Al-Hertani is a Clinical Geneticist and a Medical Biochemical Geneticist with expertise and interest in inborn errors of metabolism (IEMs) and targeted therapeutics for these IEMs Dr. Al-Hertani is currently an Attending in Genetics and Metabolism at the Boston Children’s Hospital and is the Director of the Boston Children’s Hospital Lysosomal Storage disease (BoLD) program. Most importantly to us Dr. Al-Hertani has agreed to be an advisor to Sophie’s Hope Foundation and will help provide us with guidance around clinical care and research for GSD1b. She has recently started seeing a large population of GSD patients, many of whom were previously cared for by Dr. Weinstein prior to his departure from medicine. Dr Al-Hertani will be a critical resource and advisor for our board.
Terry Derks has been certified as consultant pediatric metabolic diseases and he is appointed as associate professor at the University Medical Center Groningen, University of Groningen, the Netherlands. Since his PhD thesis defense in 2007 (title: “MCAD deficiency: clinical and laboratory studies”), he developed a special interest, both clinically and scientifically, in inherited disorders of glycogen metabolism (i.e. hepatic glycogen storage diseases) and fatty acid oxidation disorders. Dr. Derks is a trusted voice and advocate for GSD patients and their families.
Joe was first introduced to GSD1b through his best friend, Jamas LaFreniere, after his daughter Sophie was diagnosed in 2019. Joe has been with us from day one as we have navigated conversations with doctors and scientists. Joe is a graduate of Connecticut College where he graduated cum laude and earned his BA in Biochemistry, Cellular and Molecular Biology. Currently, Joe is the Director of mRNA Process Development at Strand Therapeutics in Cambridge, MA. Over his 15 + year career in the biotech industry Joe has also held strategic positions at Moderna, Percivia, and Bluebird Bio among others. Joe’s understanding of science and specifically how companies bring drugs to market for rare diseases will be an invaluable part of our foundation’s mission. Joe will be relied on to bridge the gap between research and drug development and help guide us into making proper investments in research projects. His network in the bio-tech space will be critical as we navigate the difficulties of trying to cure a rare disease. Most importantly Joe has a passion for helping people through science and specifically finding a cure for Sophie and others with GSD1b.
Jen brings a unique perspective to the CureGSD1b Advisory Board that will be critical as we navigate future treatments and the ability to commercialize them for the GSD1b community. Jen is passionate about meaningfully engaging patient communities to improve therapeutic development and the delivery of healthcare. She does this by developing a mutual understanding between stakeholders and driving collaborations around shared goals. Jen has a strong background in the pharmaceutical industry, patient advocacy non-profit, and bench research. Currently she is senior director of patient advocacy and engagement at Encoded Therapeutics, a gene regulation therapy biotech company focused on pediatric CNS diseases. Prior to joining Encoded, she was the senior director of client services at VOZ Advisors where she worked with various pharmaceutical companies on their patient focused activities. Preceding VOZ, Jen served as director and global lead of patient advocacy at bluebird bio, a biotech company developing gene therapies for severe genetic diseases and cancers. Before bluebird bio, Jen served as associate director of scientific operations at the National Brain Tumor Society, the largest nonprofit organization dedicated to the brain tumor community in the United States. Prior to finding her passion in advocacy, Jen engaged in basic research at Boston University Medical Center as a postdoctoral fellow. Jen received a BA in Psychology from the University of North Carolina Wilmington, a MA in Behavioral Neuroscience from the University of Delaware, and a PhD in Biology, Neuroscience, from the University of Victoria.